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Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy (CIDP)

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ClinicalTrials.gov Identifier: NCT02638207
Recruitment Status : Active, not recruiting
First Posted : December 23, 2015
Last Update Posted : April 26, 2019
Sponsor:
Information provided by (Responsible Party):
Octapharma

Brief Summary:
Study to evaluate the Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy

Condition or disease Intervention/treatment Phase
Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy Drug: NewGam Phase 3

Detailed Description:
Prospective, Double-blind, Randomized, Multicenter Phase III Study Evaluating Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(radiculo)neuropathy ("ProCID trial")

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 142 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Prospective, Double-blind, Randomized, Multicenter Phase III Study Evaluating Efficacy and Safety of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy
Actual Study Start Date : September 27, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: 0.5 g/kg NewGam
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Drug: NewGam
In the Dose-evaluation Phase, all patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5, 1.0 or 2.0 g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). If a patient is randomized to receive the low or medium NewGam dose, the same volume with the same infusion rate as would have been applied in case the patient would have been randomized to 2.0 g/kg NewGam will be used, thus supplemented with an authorized 0.9% w/v isotonic sodium chloride solution as appropriate and detailed in the following infusion bag split to maintain the blinding
Other Name: Panzyga

Experimental: 1.0 g/kg NewGam
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Drug: NewGam
In the Dose-evaluation Phase, all patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5, 1.0 or 2.0 g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). If a patient is randomized to receive the low or medium NewGam dose, the same volume with the same infusion rate as would have been applied in case the patient would have been randomized to 2.0 g/kg NewGam will be used, thus supplemented with an authorized 0.9% w/v isotonic sodium chloride solution as appropriate and detailed in the following infusion bag split to maintain the blinding
Other Name: Panzyga

Experimental: 2.0 g/kg NewGam
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Drug: NewGam
In the Dose-evaluation Phase, all patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5, 1.0 or 2.0 g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). If a patient is randomized to receive the low or medium NewGam dose, the same volume with the same infusion rate as would have been applied in case the patient would have been randomized to 2.0 g/kg NewGam will be used, thus supplemented with an authorized 0.9% w/v isotonic sodium chloride solution as appropriate and detailed in the following infusion bag split to maintain the blinding
Other Name: Panzyga




Primary Outcome Measures :
  1. Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score [ Time Frame: at Week 24 ]
    Efficacy - Proportion of responders in the 1.0 g/kg NewGam arm at Week 24 (Termination Visit) relative to baseline (Week 0). A responder being defined as a patient with a decrease of at least 1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score (a scale from 0 to 10, from healthy to unable to make any purposeful movements with arms and/or legs)


Secondary Outcome Measures :
  1. Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score [ Time Frame: at Week 24 ]
    Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline compared to the 1.0 g/kg arm, based on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score

  2. Grip Strength Score [ Time Frame: at Week 24 ]
    Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the grip strength (Martin Vigorimeter) using the previously published minimum clinically important difference (MCID) cut-off of 8 kilopascal (kPa)

  3. Inflammatory Rasch-built overall disability scale (I-RODS Score) [ Time Frame: at Week 24 ]
    Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the Inflammatory Rasch-built overall disability scale (I-RODS Score) using the MCID concept related to the varying standard errors (MCID-SE) as recently demonstrated

  4. Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score [ Time Frame: through study completion, an average of 2 years ]
    Time to first confirmed worsening on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale by at least 1 point from the value at baseline (Week 0)

  5. Mean Change in grip strength [ Time Frame: through study completion, an average of 2 years ]
    Mean change from baseline (Week 0) to Termination Visit in grip strength of both hands (assessed by Martin vigorimeter)

  6. Inflammatory Rasch-built overall disability scale (I-RODS) [ Time Frame: through study completion, an average of 2 years ]
    Mean change from baseline (Week 0) to Termination Visit in Inflammatory Rasch-built overall disability sum score (I-RODS using the concept of MCID-SE as recently reported; Appendix 1) and number of improvers

  7. Right sided motor nerves [ Time Frame: through study completion, an average of 2 years ]
    Mean change from baseline (Week 0) to Termination Visit in sum of the distal evoked amplitude of 4 right sided and 4 left sided motor nerves (peroneal, tibial, ulnar and median)

  8. Mean Change in pain intensity numerical rating scale (PI-NRS Scale) [ Time Frame: through study completion, an average of 2 years ]
    Mean change from baseline (Week 0) to Termination Visit in Pain Intensity Numeric Rating Scale (PI-NRS)

  9. Worsening on the Inflammatory Rasch-built overall disability scale (I-RODS Scale) [ Time Frame: through study completion, an average of 2 years ]
    Time to first confirmed worsening on the I-RODS scale

  10. 1 point decrease in the INCAT disability score [ Time Frame: through study completion, an average of 2 years ]
    Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score

  11. decrease in Inflammatory Rasch-built overall disability scale (I-RODS Scale) [ Time Frame: through study completion, an average of 2 years ]
    Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with diagnosis of definite or probable Chronic inflammatory demyelinating polyneuropathy (CIDP) according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) Guideline 2010 [van den Bergh et al., 2010]; including patients with Multifocal Acquired Demyelinating Sensory And Motor Neuropathy (MADSAM) or pure motor Chronic inflammatory demyelinating polyneuropathy (CIDP )
  2. Patients currently depending on treatment with immunoglobulins or corticosteroids
  3. Patients with active disease, i.e. not being in remission, who are progressive or relapsing prior to trial start or during the Wash-out Phase
  4. Weakness of at least 2 limbs
  5. >18 to <80 years of age
  6. Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score between 2 and 9 (with a score of 2 coming exclusively from leg disability)
  7. Voluntarily given, fully informed written consent obtained from patient before any study-related procedures are conducted

Exclusion Criteria:

  1. Unifocal forms of Chronic inflammatory demyelinating polyneuropathy (CIDP)
  2. Pure sensory Chronic inflammatory demyelinating polyneuropathy (CIDP)
  3. Multifocal motor neuropathy (MMN) with conduction block [van den Bergh et al., 2010]
  4. Patients who previously failed immunoglobulin treatment
  5. Treatment with immunomodulatory/suppressive agents (cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil or azathioprine) during the six months prior to baseline visit
  6. Patients on or treated with rituximab, alemtuzumab, cyclophosphamide, or other intensive chemotherapeutic regimens, previous lymphoid irradiation or stem cell transplantation during the 12 months prior to baseline visit
  7. Respiratory impairment requiring mechanical ventilation
  8. Myelopathy or evidence of central nervous system demyelination or significant persisting neurological deficits from stroke, or central nervous system (CNS) trauma
  9. Clinical evidence of peripheral neuropathy from another cause such as

    1. connective tissue disease or systemic lupus erythematosus (SLE)
    2. HIV infection, hepatitis, Lyme disease
    3. cancer (with the exception of basal cell skin cancer)
    4. IgM paraproteinemia with anti-myelin associated glycoprotein antibodies
  10. Diabetic neuropathy
  11. Cardiac insufficiency (New York Heart Association [NYHA] III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease
  12. Severe liver disease (ALAT 3x > normal value)
  13. Severe kidney disease (creatinine 1.5x > normal value)
  14. Hepatitis B, hepatitis C or HIV infection
  15. Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever; patients with susceptibility to embolism or deep vein thrombosis (DVT)
  16. Body mass index (BMI) ≥40 kg/m2
  17. Patients with uncompensated hypothyroidism (abnormally high Thyroid-Stimulating Hormone [TSH] and abnormally low Thyroxine [T4]) or known vitamin B12 deficiency if patients don't receive adequate substitution therapy
  18. Medical conditions whose symptoms and effects could alter protein catabolism and/or Immunoglobulin G (IgG) utilization (e.g. protein-losing enteropathies, nephrotic syndrome)
  19. Known Immunoglobulin A (IgA) deficiency with antibodies to Immunoglobulin A (IgA)
  20. History of severe hypersensitivity, e.g. anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of NewGam
  21. Known blood hyperviscosity, or other hypercoagulable states
  22. Use of other blood or plasma-derived products within three months prior to Visit 2
  23. Patients with a past or present history of drug abuse or alcohol abuse within the preceding five years prior to baseline visit
  24. Patients unable or unwilling to understand or comply with the study protocol
  25. Participation in another interventional clinical study with investigational medicinal product (IMP) treatment currently or during the three months prior to Visit 2
  26. Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method (such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner) while on study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02638207


  Show 37 Study Locations
Sponsors and Collaborators
Octapharma
Investigators
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Study Director: Wolfgang Frenzel, MD Octapharma

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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02638207     History of Changes
Other Study ID Numbers: NGAM-08
First Posted: December 23, 2015    Key Record Dates
Last Update Posted: April 26, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Octapharma:
Chronic Inflammatory Demyelinating Poly Neuropathy (CIDP)

Additional relevant MeSH terms:
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Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases